School of Medicine, University of California, San Diego, La Jolla, CA, USA.
Department of Surgery, Division of Colon and Rectal Surgery, University of California, San Diego Health Systems, La Jolla, CA, 92093-0987, USA.
Ann Surg Oncol. 2022 Mar;29(3):1894-1907. doi: 10.1245/s10434-021-10576-z. Epub 2021 Sep 16.
Watch and wait (WW) protocols have gained increasing popularity for patients diagnosed with locally advanced rectal cancer and presumed complete clinical response after neoadjuvant chemoradiation. While studies have demonstrated comparable survival and recurrence rates between WW and radical surgery, the decision to undergo surgery has significant effects on patient quality of life. We sought to conduct a cost-effectiveness analysis comparing WW with abdominoperineal resection (APR) and low anterior resection (LAR) among patients with stage II/III rectal cancer.
In this comparative-effectiveness study, we built Markov microsimulation models to simulate disease progression, death, costs, and quality-adjusted life-years (QALYs) for WW or APR/LAR. We assessed cost effectiveness using the incremental cost-effectiveness ratio (ICER), with ICERs under $100,000/QALY considered cost effective. Probabilities of disease progression, death, and health utilities were extracted from published, peer-reviewed literature. We assessed costs from the payer perspective.
WW dominated both LAR and APR at a willingness to pay (WTP) threshold of $100,000. Our model was most sensitive to rates of distant recurrence and regrowth after WW. Probabilistic sensitivity analysis demonstrated that WW was the dominant strategy over both APR and LAR over 100% of iterations across a range of WTP thresholds from $0-250,000.
Our study suggests WW could reduce overall costs and increase effectiveness compared with either LAR or APR. Additional clinical research is needed to confirm the clinical efficacy and cost effectiveness of WW compared with surgery in rectal cancer.
对于接受新辅助放化疗后诊断为局部晚期直肠癌且临床完全缓解的患者，观察等待（WW）方案越来越受欢迎。虽然研究表明 WW 与根治性手术之间的生存率和复发率相当，但手术的决定对患者的生活质量有重大影响。我们旨在进行一项成本效益分析，比较 WW 与腹会阴切除术（APR）和低位前切除术（LAR）在 II/III 期直肠癌患者中的应用。
在这项比较有效性研究中，我们构建了 Markov 微模拟模型，以模拟 WW 或 APR/LAR 患者的疾病进展、死亡、成本和质量调整生命年（QALY）。我们使用增量成本效益比（ICER）评估成本效益，ICER 低于 100,000 美元/QALY 被认为具有成本效益。疾病进展、死亡和健康效用的概率从已发表的同行评议文献中提取。我们从支付者的角度评估成本。
在 100,000 美元的支付意愿（WTP）阈值下，WW 优于 LAR 和 APR。我们的模型对 WW 后远处复发和再生长的发生率最为敏感。概率敏感性分析表明，在 0-250,000 美元的一系列 WTP 阈值范围内，WW 在 100%的迭代中都是优于 APR 和 LAR 的主导策略。
我们的研究表明，与 LAR 或 APR 相比，WW 可能会降低总成本并提高效果。需要进一步的临床研究来证实 WW 在直肠癌中与手术相比的临床疗效和成本效益。
